Rethinking the nonprofit foundation: an emerging niche in the rare disease ecosystem

In recent years, medical foundations have become increasingly influential, and now play an instrumental and integral role in the research and development of their disease area of interest. While some foundations have directly invested in taking drug candidates to the clinic, others have focused on creating specific tools for accelerating the identification and development of effective treatments. Here, we describe a new model, developed by the Children's Tumor Foundation (CTF), by which foundations may play a role in the rare disease ecosystem. On the one hand, the CTF uses its position to build bridges between academic scientists, biotech and pharmaceutical companies, and patients, to accelerate the development of treatments that really matter most to patients. On the other hand, it acts as a niche investor to fund an integrated platform for critical R&D endeavors—including elements such as a patient registry, biobank, and open data platforms—which smoothen the transition from basic discovery to clinical benefit. Currently, the Children's Tumor Foundation is launching a call to top finance experts to collaborate on building an innovative model that will guarantee long‐term sustainability of this integrated platform (Fig 1).